Trial Outcomes & Findings for Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (NCT NCT00227266)

NCT ID: NCT00227266

Last Updated: 2025-03-19

Results Overview

Participants will have labs drawn regularly to maintain appropriate dosing and monitor liver function

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

94 participants

Primary outcome timeframe

-4 wks, 0, 2 wks, 3 mo, 6 mo, 9 mo, 12 mo for safety labs; throughout for AEs

Results posted on

2025-03-19

Participant Flow

Subject's were recruited during the periods of September 2005 to September 2006 across the United States.

Participant milestones

Participant milestones
Measure	Cohort 1a Sitters Placebo Then Treatment Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Overall Study STARTED	31	30	33
Overall Study COMPLETED	30	30	29
Overall Study NOT COMPLETED	1	0	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1a Sitters Placebo Then Treatment Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 2 Standers and Walkers - Treatment Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Overall Study Withdrawal by Subject	0	1
Overall Study Protocol Violation	1	1
Overall Study Excessive weight gain	0	2

Baseline Characteristics

Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1a Sitters Placebo Then Treatment n=31 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment n=30 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment n=33 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Total n=94 Participants Total of all reporting groups
Age, Categorical <=18 years	31 Participants n=5 Participants	30 Participants n=7 Participants	33 Participants n=5 Participants	94 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	4.4 years STANDARD_DEVIATION 1.9 • n=5 Participants	4.3 years STANDARD_DEVIATION 2.1 • n=7 Participants	7.3 years STANDARD_DEVIATION 3.7 • n=5 Participants	5.4 years STANDARD_DEVIATION 3.0 • n=4 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	17 Participants n=7 Participants	11 Participants n=5 Participants	39 Participants n=4 Participants
Sex: Female, Male Male	20 Participants n=5 Participants	13 Participants n=7 Participants	22 Participants n=5 Participants	55 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	3 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	29 Participants n=5 Participants	27 Participants n=7 Participants	30 Participants n=5 Participants	86 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	5 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	4 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) White	26 Participants n=5 Participants	25 Participants n=7 Participants	29 Participants n=5 Participants	80 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	9 Participants n=4 Participants
Region of Enrollment United States	25 participants n=5 Participants	26 participants n=7 Participants	29 participants n=5 Participants	80 participants n=4 Participants
Region of Enrollment Canada	6 participants n=5 Participants	4 participants n=7 Participants	4 participants n=5 Participants	14 participants n=4 Participants

PRIMARY outcome

Timeframe: -4 wks, 0, 2 wks, 3 mo, 6 mo, 9 mo, 12 mo for safety labs; throughout for AEs

Participants will have labs drawn regularly to maintain appropriate dosing and monitor liver function

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: -4wks, 0, 3 mo, 6 mo, 12 mo

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 0 months, 6 months

Population: Analysis only pertains to cohort 1a and 1b.

Comparison of Modified Hammersmith Change from baseline to 6 months. Scores range from 0 to 40. A higher score indicates a better outcome. This scale is used to assess gross motor abilities of non-ambulant children with SMA in multiple research trials as well as in clinical settings.

Outcome measures

Outcome measures
Measure	Cohort 1a Sitters Placebo Then Treatment n=31 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment n=30 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Modified Hammersmith Change From Baseline to 6 Months Baseline visit (0 weeks)	20.0 Score Standard Deviation 9.3	16.6 Score Standard Deviation 8.7	—
Modified Hammersmith Change From Baseline to 6 Months 6 Month visit (V2)	20.6 Score Standard Deviation 8.1	16.8 Score Standard Deviation 7.9	—
Modified Hammersmith Change From Baseline to 6 Months Change from Baseline	0.6 Score Standard Deviation 3.98	0.2 Score Standard Deviation 2.88	—

SECONDARY outcome

Timeframe: -4wks or 0, 3 mo, 6 mo, 12 mo

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: -4wks, 0, 3mo, 6mo, 12mo

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)

The maximum Compound Motor Action Potential (CMAP) is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This is done multiple times, the outcome used is the highest peak, or response observed.

Outcome measures

Outcome measures
Measure	Cohort 1a Sitters Placebo Then Treatment n=23 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment n=21 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment n=24 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Max CMAP Amplitude (Mean) Baseline	2.28 mV Standard Deviation 1.55	2.93 mV Standard Deviation 1.56	5.52 mV Standard Deviation 2.56
Max CMAP Amplitude (Mean) 6 months	2.32 mV Standard Deviation 1.75	2.37 mV Standard Deviation 1.82	6.56 mV Standard Deviation 2.99

SECONDARY outcome

Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)

Outcome measures

Outcome measures
Measure	Cohort 1a Sitters Placebo Then Treatment n=23 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment n=21 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment n=24 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Max CMAP Amplitude Median Baseline	1.91 mV Interval 0.5 to 7.66	2.2 mV Interval 0.5 to 6.66	5.3 mV Interval 1.2 to 10.42
Max CMAP Amplitude Median 6 months	1.44 mV Interval 0.5 to 6.14	1.8 mV Interval 0.3 to 7.81	5.85 mV Interval 1.5 to 12.2

SECONDARY outcome

Timeframe: -4 wks, 0, 3 mo, 6 mo, 12 mo

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: -4 wks, 0, 3mo, 6mo, 12mo

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: -4 wks, 0, 3mo, 6mo, 12mo

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 0, 6mo, 12mo

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)

The maximum Compound Motor Action Potential (CMAP) area is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This procedure is repeated multiple times. The maximum area is the response that results in the largest area under the response curve.

Outcome measures

Outcome measures
Measure	Cohort 1a Sitters Placebo Then Treatment n=23 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment n=21 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment n=24 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Max CMAP Area (Mean) 6 months	5.28 mVms Standard Deviation 4.49	5.26 mVms Standard Deviation 4.65	16.26 mVms Standard Deviation 7.13
Max CMAP Area (Mean) Baseline	5.46 mVms Standard Deviation 5.03	5.45 mVms Standard Deviation 4.23	14.85 mVms Standard Deviation 7.68

SECONDARY outcome

Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)

Outcome measures

Outcome measures
Measure	Cohort 1a Sitters Placebo Then Treatment n=23 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment n=21 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment n=24 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Max CMAP Area (Median) Baseline	3.6 mVms Interval 0.7 to 19.71	4.6 mVms Interval 1.3 to 16.78	13.65 mVms Interval 2.6 to 38.29
Max CMAP Area (Median) 6 months	3.74 mVms Interval 0.6 to 16.13	3.4 mVms Interval 0.6 to 18.81	16.85 mVms Interval 3.7 to 29.1

POST_HOC outcome

Timeframe: 1 month prior to enrollment, at enrollment (0 months)

Population: Analysis was determined per protocol

Baseline Modified Hammersmith Extend testing. The baseline test is the score they receive during their screening visits. This scale ranges from 0 to 56. A higher score indicates a better outcome. This scale is used to assess gross motor abilities of children with SMA in multiple research trials as well as in clinical settings.

Outcome measures

Outcome measures
Measure	Cohort 1a Sitters Placebo Then Treatment n=33 Participants Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Modified Hammersmith Extend Baseline Modified Hammersmith Extend at S1 (-4 weeks)	47.0 Score Interval 29.0 to 56.0	—	—
Modified Hammersmith Extend Baseline Modified Hammersmith Extend at S2 (0 weeks)	48.3 Score Interval 36.0 to 56.0	—	—

Adverse Events

Cohort 1a Sitters Placebo Then Treatment

Serious events: 1 serious events

Other events: 18 other events

Deaths: 0 deaths

Cohort 1b Sitters Treatment

Serious events: 4 serious events

Other events: 23 other events

Deaths: 0 deaths

Cohort 2 Standers and Walkers - Treatment

Serious events: 4 serious events

Other events: 28 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1a Sitters Placebo Then Treatment n=31 participants at risk Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.	Cohort 1b Sitters Treatment n=30 participants at risk Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.	Cohort 2 Standers and Walkers - Treatment n=33 participants at risk Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.3% 1/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	0.00% 0/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Metabolism and nutrition disorders Dehydration	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.3% 1/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	0.00% 0/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Gastrointestinal disorders Diarrhea	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	0.00% 0/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	6.1% 2/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
General disorders General Disorder	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.3% 1/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	0.00% 0/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Respiratory, thoracic and mediastinal disorders Pneumonia	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	6.7% 2/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.0% 1/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Infections and infestations Pneumonitis	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.3% 1/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	0.00% 0/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Skin and subcutaneous tissue disorders Skin Rash	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	0.00% 0/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.0% 1/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Respiratory, thoracic and mediastinal disorders Tachyponea	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.3% 1/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.0% 1/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Infections and infestations Upper respiratory infection	3.2% 1/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	0.00% 0/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.0% 1/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Gastrointestinal disorders Vomiting	0.00% 0/31 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.3% 1/30 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months	3.0% 1/33 • Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months

Other adverse events

Additional Information

Sandra Reyna, M.D.

University of Utah

Phone: 801-581-3551

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place